Midwest Regional Forum. Work Smarter Not Harder Leveraging Prior Knowledge to Accelerate CMC Programs

Transcription

1 Midwest Regional Forum Work Smarter Not Harder Leveraging Prior Knowledge to Accelerate CMC Programs Tuesday, October 23, 2018 Loews Chicago O Hare Hotel Rosemont, IL Meeting Co-chairs: Sarah Demmon, Eli Lilly and Company Robert Seevers, Pearl Pathways

2 The program committee gratefully acknowledges the following program partners and exhibitors for their generous support of the CASSS Midwest Regional Forum Strategic Gold Program Partners Eli Lilly and Company Pfizer, Inc. Exhibitor Partners Catalent Pharma Solutions Eurofins Lancaster Laboratories

3 Acknowledgements Special thanks to the program committee who helped develop this program! Program Committee: Sergey Arzhantsev, CDER, FDA Leslie Bloom, Pfizer, Inc. Michael Boyne, COUR Pharmaceuticals Development Company Holly Cargill, Pfizer, Inc. James Carroll, Pfizer, Inc. Sarah Demmon, Eli Lilly and Company JR Dobbins, Eli Lilly and Company Don Eisenhauer, AbbVie Inc. Will Hatcher, Catalent Pharma Solutions Julie Heflin, AbbVie Inc. Michelle Lytle, Eli Lilly and Company Khalid Mahmood, Eli Lilly and Company Lisa McDermott, Millipore Sigma Edwin Moore, University of Illinois Ned Mozier, Pfizer, Inc. Lesley Redfern, AbbVie Inc. Robert Seevers, Pearl Pathways Qin Sheng, Eli Lilly and Company Jason Starkey, Pfizer, Inc. CASSS Staff: Karen A. Bertani, CMP, Director, Global Engagement and Knowledge Sharing Noelle Atkins, Administrative Coordinator Stephanie L. Flores, CAE, Executive Director Julie Fowle, Program Planning and Event Specialist Anna Lingel, CMP, Exhibitor Relations and Technology Specialist Renee Olson, Senior Program Manager Catherine Federle Stewart, Finance Manager

4 Preliminary Scientific Program Summary 18:00 20:00 Welcome Reception 08:00 15:30 Registration 08:00 09:00 Breakfast Monday, October 22, 2018 Tuesday, October 23, :00 09:15 CASSS Welcome and Introductory Comments Sarah Demmon, Eli Lilly and Company 09:15 09:30 Welcome to the Midwest Regional Forum Sarah Demmon, Eli Lilly and Company 09:30 10:00 A Regulatory Perspective on the Use of Prior Knowledge to Accelerate CMC Development Brian Roelofs, CDER, FDA 10:00 10:15 Networking Break. 10:15 10:45 Leveraging Prior Knowledge during Biotherapeutics Development Margaret Ruesch, Pfizer, Inc. 10:45 11:30 Leveraging Platform Knowledge to Estimate Bioprocess Variability by Applying Bayesian Methodology Jinxin (Jerry) Gao, Eli Lilly and Company Leveraging Prior Knowledge to Accelerate CMC Programs Elisabeth Krug, Eli Lilly and Company 11:30 12:15 Panel Discussion Panel Moderator: Sarah Demmon, Eli Lilly and Company Panel Members: Brian Roelofs, CDER, FDA Jerry Gao, Eli Lilly and Company Elisabeth Krug, Eli Lilly and Company Margaret Ruesch, Pfizer, Inc.

5 12:15 13:30 Career Development Roundtable / Lunch 13:30 14:30 Case Study: Gaining Agreement with FDA on a Simplified, Rational Package and Shipping Qualification Program Paul Harber, Parenteral Supply Chain, LLC 14:30 14:45 Networking Break 14:45 15:20 Case Study continued ADCs Lisa McDermott, Millipore Sigma 15:20 15:30 Closing Remarks Robert Seevers, Pearl Pathways

6 Program Abstract Work Smarter Not Harder - Leveraging Prior Knowledge to Accelerate CMC Programs CMC programs are under intense pressure to deliver commercial ready manufacturing processes early in the project life cycle. In this session, we will explore strategies to speed delivery of material to the clinic for FIH trials by using prior knowledge and project platform approaches. Speakers will also discuss strategies about streamlining development studies to lock down process decisions earlier in the development cycle. Finally, we will discuss a case study to explore how companies are using prior knowledge. The afternoon will feature two interactive case studies showing how risk-based approaches have been applied, the first on data needed to support shipping of temperature sensitive biologics, and the second on the development of antibody-drug conjugates (ADCs.)

7 Presenter Abstracts A Regulatory Perspective on the Use of Prior Knowledge to Accelerate CMC Development Brian Roelofs, CDER, FDA, Silver Spring, MD USA Approaches to product development differ from product to product, even within the same company. Minimally, product CMC development should include identification of critical quality attributes and definition of the appropriateness of the manufacturing process and control strategies to ensure process performance and product quality. Prior knowledge is a tool that can be used to enhance, inform, and potentially accelerate development of biological products. Manufacturing processes, formulations, presentations, and control strategies can all be supported with prior knowledge. The extent to which prior knowledge can be leveraged depends on the justification provided with each product. This presentation will begin with an overview of the concept of prior knowledge. From this framework, we will discuss the potential applications of prior knowledge described in current regulatory guidance such as ICH, FDA guidances for industry, and related documents. In addition, the presentation will describe the Agency s expectations regarding the use of prior knowledge as supportive information for product and process control strategies in applications. Examples of both the opportunities and the limitations of using this type of information to support development will also be described.

8 Leveraging Prior Knowledge during Biotherapeutics Development Margaret Ruesch, Pfizer, Inc., Andover, MA USA Prior knowledge provides an important starting point for process and analytical product development for biotherapeutics. Prior knowledge is a broad term that encompasses experience with similar products, published literature, and internal technical data. Use of prior knowledge is aligned with regulatory expectations; brings medicines to patients faster; and ensures appropriate focus for process and analytical development organizations. This talk will discuss examples of prior knowledge informing quality attribute assessment, commercial specification setting, and new modality analytical development.

9 Leveraging Platform Knowledge to Estimate Bioprocess Variability by Applying Bayesian Methodology Jinxin (Jerry) Gao, Eli Lilly and Company, Indianapolis, IN USA Bioprocess variability is the variation that occurs during the biopharmaceutical manufacturing process. This variability is an accumulation of many different sources of variation, such as raw material, measurement, and sampling. We will discuss how to estimate bioprocess variability based on platform knowledge from multiple molecules and multiple manufacturing sites.

10 Leveraging Prior Knowledge to Accelerate CMC Programs Elisabeth Krug, Eli Lilly and Company, Indianapolis, IN USA Development timelines decrease, but experience with the molecules to be developed increases. Shouldn t we be able to apply prior knowledge to aid in accelerating development? From a general development perspective, one could think of phase-appropriate approaches to the understanding of the individual molecule as a lever. From an analytical perspective, there are opportunities to take advantage of platform methods that support general process-related impurity evaluations and first-to-try conditions for product-related substances. This talk will illustrate approaches taken at Eli Lilly and Company.

11 Career Development Roundtable TOPIC: FACILITATORS: Career Development Transferable Skills Michael Boyne, COUR Pharmaceuticals Development Company Will Hatcher, Catalent Pharma Solutions SCOPE: We will be discussing career development within the biotech and pharma industries. We will explore myths regarding the number of years one has to acquire to attain a position of different area or discipline in the industry. We will also discuss transferable skills, management experience, and strategic skill sets. QUESTIONS FOR DISCUSSION: 1. Is it important to have a degree in the field that you are pursuing? a. For example, if the goal is to be a Director of Analytics, does one need degree in chemistry or biology? Does one require a Ph.D.? b. Is this the case for all careers? For example, does this hold true for regulatory affairs or project management? 2. How does one move from technical jobs to regulatory affairs, project management, quality assurance, and vice versa, etc.? a. What are transferable skills in the industry? b. How can you start thinking more cross-functionally? c. How can you think more strategically? 3. Is it better to have a more diverse background or focus on one field for many years to be successful? a. How can one gain more experience in one discipline yet still stay in their current position?

12 Case Study Abstract Gaining Agreement with FDA on a Simplified, Rational Package and Shipping Qualification Program Paul Harber, Parenteral Supply Chain, LLC, Indianapolis, IN USA A rational package and shipping qualification program refers to a combination of work streams. A successful program utilizes a risk-based plan combining product knowledge and process control. It is impossible to have any alignment discussion with the FDA if these work streams are not in place, well defined and actively coordinated. Recently the FDA has focused on one of these work streams in particular, issuing Information Requests (IR) to numerous applicants. Well-defined work streams will help gain alignment with external regulators. Internally, it will identify the tasks necessary for a successful filing. This presentation will identify the work streams, present the typical IR recently issued by the FDA and introduce a methodology for filing the PV Shipping/Distribution sections of a BLA. Discussions of this type may be new for innovators, but they are becoming necessary to have with the regulatory agencies well before filing.